Clinical Trial

Trial Name

What is the trial about?

The purpose of this study is to learn more about how patients with achondroplasia grow.

Who can participate?

To be in this study your child must:

have achondroplasia

be 13½ years old or younger

be willing and able to perform all study procedures as physically possible

be able to walk and stand if they are age 2 or older

Your child cannot be in this study if s/he:

has a disease or condition not allowed such as high blood pressure, heart disease, insulin-requiring diabetes mellitus

is taking medication that is not allowed, such as a growth hormone or oral steroid

The study staff will discuss a more complete list of diseases and medications that would exclude your child from the study with you.

What is involved?

The study will last from about 6 months to about 5 years, including between 3 and 22 study visits. After about 6 months (at least 3 visits), you may be offered the option to enroll your child in a different study, and your child will no longer participate in this study. Otherwise, your child may continue in this study until the end of the study (about 22 visits over up to 5 years).

New things are learned in clinical studies by doing tests. There are tests and procedures for this study that your child will be asked to do and some of these are part of their normal care, such as having measurements and vital signs taken and having physical exams. For this study, there will be more measurements taken than usual.

The study doctor will check to see whether your child can be in the study by doing the screening procedures described below. After the screening, if the doctor confirms that your child can participate and you/your child choose to take part, the tests and procedures described below will be completed.

Medical History: You will be asked about your child’s medical history including diseases, medications, hospital admissions, and any medical or surgical procedures that your child has had in the past. You may also be asked about your child’s previous growth measurements and ACH-related tests and procedures.

Physical Examination: Your child’s body will be examined to determine their physical health. You or your child may also be asked questions about your child’s current health status.

Vital Signs: Your child’s vital signs such as heart rate, breathing rate, and blood pressure will be taken. Your child may or may not have an optional test called pulse oximetry. This test measures the amount of oxygen that is carried in blood.

Vitamin D: A blood sample will be taken from a needle placed in your child’s arm. The vitamin D test will help the study doctor to know whether bone weakness or other issues are due to a vitamin D problem. Depending on the results, the study doctor may discuss additional tests or treatment with you that are not part of the study. About one to two teaspoons or 5-10 mL of blood will be taken.

Alkaline phosphatase (ALP): ALP levels may be related to bone growth changes. The ALP test may help to define a range of alkaline phosphatase levels that are seen in children with ACH. About half a teaspoon of blood will be taken.

Measurements: Your child’s weight and height will be measured. Measurements of your child’s arms, head, legs, waist, body, chest, hands and feet will also be taken a few times and in a few different ways at each study visit. Your child will be asked to stay in one position while each measurement takes place. Your child’s body mass index will be calculated from their height and weight measurements.

Tanner Stage of sexual development: If your child is aged 5 to 13½, the study staff will determine your child’s current stage of development based on things like breast size and development of pubic hair.

Adverse Events, Medications, and ACH Related Tests and Symptoms: You will be asked about your child’s health and medications since the previous visit. You will also be asked about any ACH-related tests and symptoms.